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Donor Stem Cell Transplant With or Without Chemotherapy in Treating Children With Primary Myelodysplastic Syndrome

The recruitment status of this study is unknown because the information has not been verified recently.
Verified July 2007 by National Cancer Institute (NCI).
Recruitment status was  Active, not recruiting
Sponsor:
Information provided by:
National Cancer Institute (NCI)
ClinicalTrials.gov Identifier:
NCT00047268
First received: October 3, 2002
Last updated: May 14, 2011
Last verified: July 2007
History of Changes
  Purpose

RATIONALE: Giving chemotherapy before a donor stem cell transplant helps stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. It is not yet known whether donor stem cell transplant is more effective with or without chemotherapy in treating primary myelodysplastic syndrome.

PURPOSE: This phase III trial is studying how well donor stem cell transplant given with chemotherapy works and compares it with donor stem cell transplant without chemotherapy in treating children with primary myelodysplastic syndrome.


Condition Intervention Phase
Leukemia
Myelodysplastic/Myeloproliferative Neoplasms
 Drug: cytarabine
Drug: mercaptopurine
Other: laboratory biomarker analysis
Procedure: allogeneic bone marrow transplantation
Procedure: biopsy
Procedure: peripheral blood stem cell transplantation
 Phase 3

Study Type: Interventional
Study Design: Masking: Open Label
Primary Purpose: Diagnostic
Official Title: Prospective Study of the Diagnosis and Treatment of Myelodysplastic Syndromes (MDS) in Childhood

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by National Cancer Institute (NCI):

Primary Outcome Measures:
  • Patient numbers in the different FAB subtypes [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Survival [ Designated as safety issue: No ]
  • Event-free survival [ Designated as safety issue: No ]

Study Start Date: July 1998
Detailed Description:

OBJECTIVES:

  • Determine, by a standard approach, the frequency of different FAB subtypes in children with primary myelodysplastic syndromes.
  • Determine the frequency of cytogenetic and molecular abnormalities in these patients.
  • Determine the survival of patients treated with allogeneic stem cell transplantation with or without induction chemotherapy.
  • Determine the rate of complete remission in patients treated with these regimens.
  • Determine the event-free survival of patients treated with these regimens.
  • Determine the relapse rate, morbidity, and mortality of patients treated with these regimens.
  • Determine different subsets of patients who benefit from these regimens.

OUTLINE: This is a multicenter study. Patients are stratified according to FAB subtype (refractory anemia (RA) or RA with ringed sideroblasts (RARS) vs RA with excess blasts (RAEB) vs RAEB in transformation (RAEB-t) vs juvenile myelomonocytic leukemia (JMML)).

Patients undergo complete medical and physical examination. Patients are screened for the following aberrations: -7, +8, +21, t(8;21), t(15;17), and inv(16). Smears of peripheral blood and bone marrow, as well as bone marrow biopsies and all cytogenetic and molecular studies performed on blood or bone marrow, are evaluated by a panel of international experts.

Patients with progressive RA or RARS undergo allogeneic stem cell transplantation (ASCT) according to EWOG-MDS SCT studies. Patients with stable RA or RARS wait for an optimal donor before undergoing ASCT. Patients with RAEB with fewer than 15% bone marrow blasts undergo ASCT. Patients with RAEB with at least 15% bone marrow blasts and patients with RAEB-t with fewer than 30% bone marrow blasts receive standard acute myeloid leukemia (AML) induction therapy and then undergo ASCT. Patients with RAEB-t with at least 30% bone marrow blasts are considered for standard AML induction therapy.

Patients with advanced JMML undergo evaluation for splenectomy and receive chemotherapy with mercaptopurine and cytarabine every 3-4 weeks (for 1-4 doses). Patients then undergo ASCT.

Patients are followed every 6 months.

PROJECTED ACCRUAL: Not specified

  Eligibility

Ages Eligible for Study:   up to 18 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

DISEASE CHARACTERISTICS:

  • Morphologically confirmed primary myelodysplastic syndromes (MDS)

    • Diagnosed between July 1, 1998 and June 30, 2002
  • No prior aplastic anemia

  • No prior congenital bone marrow failure syndrome, such as:

    • Fanconi's anemia
    • Kostmann syndrome
    • Shwachman syndrome
    • Dyskeratosis congenital
    • Amegakaryocytic thrombocytopenia
    • Diamond-Blackfan anemia
  • No Down syndrome

  • None of the following cytogenetic or molecular abnormalities:

    • t(8;21)(q22;q22)
    • t(15;17)(q22;q12)
    • inv(16)(p13;q22)
  • No typical clinical and cytogenetic features of acute myeloid leukemia FAB M7 (i.e., acute megakaryocytic leukemia) with fewer than 30% blasts in bone marrow or peripheral blood

PATIENT CHARACTERISTICS:

Age

  • Under 19

Performance status

  • Not specified

Life expectancy

  • Not specified

Hematopoietic

  • See Disease Characteristics

Hepatic

  • Not specified

Renal

  • Not specified

Other

  • No other concurrent illness that would preclude study

PRIOR CONCURRENT THERAPY:

Biologic therapy

  • Not specified

Chemotherapy

  • No prior chemotherapy for MDS

Endocrine therapy

  • Not specified

Radiotherapy

  • No prior radiotherapy for MDS

Surgery

  • Not specified
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00047268

Locations
Germany
Universitaetskinderklinik - Universitaetsklinikum Freiburg
Freiburg, Germany, D-79106
Sponsors and Collaborators
European Working Group of MDS in Childhood
Investigators
Study Chair: Charlotte Niemeyer, MD Universitaetskinderklinik - Universitaetsklinikum Freiburg
  More Information

Additional Information:
Clinical trial summary from the National Cancer Institute's PDQ® database  This link exits the ClinicalTrials.gov site

No publications provided by National Cancer Institute (NCI)

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

ClinicalTrials.gov Identifier: NCT00047268     History of Changes
Other Study ID Numbers: CDR0000257581, EWOG-MDS-98, EU-20218
Study First Received: October 3, 2002
Last Updated: May 14, 2011
Health Authority: United States: Federal Government

Keywords provided by National Cancer Institute (NCI):
juvenile myelomonocytic leukemia
childhood myelodysplastic syndromes
atypical chronic myeloid leukemia, BCR-ABL1 negative
myelodysplastic/myeloproliferative neoplasm, unclassifiable
chronic myelomonocytic leukemia

Additional relevant MeSH terms:
Neoplasms
Leukemia
Myelodysplastic Syndromes
Preleukemia
Myeloproliferative Disorders
Myelodysplastic-Myeloproliferative Diseases
Neoplasms by Histologic Type
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
6-Mercaptopurine
Cytarabine
Antimetabolites
 Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Antimetabolites, Antineoplastic
Antineoplastic Agents
Therapeutic Uses
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Nucleic Acid Synthesis Inhibitors
Enzyme Inhibitors
Antiviral Agents
Anti-Infective Agents

ClinicalTrials.gov processed this record on May 22, 2013